REAL-WORLD REASONS FOR STEROID SWITCHING AND ASSOCIATED CLINICAL OUTCOMES AMONG PATIENTS WITH DYSTROPHINOPATHIES: NATIONWIDE REVIEW OF MEDICAL RECORDS

Clinical Management
94
Jessica Marden MPH, Claudio Santos MD, Brian Pfister PhD, Henry Lane BA, Michael Somma BA, Jing Zhao PhD, James Signorovitch PhD, Julie Parsons MD, Susan Apkon MD
1. Analysis Group, Inc., 2. PTC Therapeutics, 3. PTC Therapeutics - South Plainfield, NJ, 4. Analysis Group, Inc., 5. Group Analysis Inc, 6. Group Analysis Inc, 7. Analysis Group, Inc., 8. Children Hospital-Colorado, 9. Children Hospital-Colorado

BACKGROUND: Corticosteroid treatment with prednisone/prednisolone or deflazacort is the standard of care for dystrophinopathies. Improved understanding of real-world switching is needed following deflazacort approval.
OBJECTIVE: To describe reasons for switching from prednisone/prednisolone to deflazacort, and clinical outcomes, among US patients with dystrophinopathies.
METHODS: Chart review conducted by 55 neurologists for patients with dystrophinopathy who switched from prednisone to deflazacort between February 2017 and December 2018.
RESULTS: Charts were reviewed for 102 males in community (49%) and academic (51%) settings. Seventy had Duchenne muscular dystrophy; mean ± SD age 11.6 ± 10.4 years and 80% ambulatory at switch. Thirty-two had Becker’s muscular dystrophy; mean age 21.2 ± 12.5 years and 81% ambulatory at switch. Average treatment duration was 3.3 years for prednisone and 6 months for deflazacort prior to the chart extraction. Physicians ranked “desire to slow disease progression” and “tolerability issues” as primary reasons for switching in 78% and 57%, respectively. Switching was reported as “very” or “somewhat” effective at addressing primary reasons in 95% of patients. Commonly recorded adverse effects during prednisone and deflazacort treatment included weight gain (38% and 15%), Cushingoid appearance (26% and 10%), increased appetite (20% and 6%), central obesity (10% and 3%), and fluid retention (9% and 2%); hirsutism occurred for 2% and 4%. Findings were similar in the subgroup (n=62) with ≥ 3 months deflazacort. Among 34 patients with recorded Global Impression of Illness Severity, 8 (24%) improved, 24 (70%) remained the same, and 2 (6%) worsened after switching.
CONCLUSIONS: In this real-world study, the majority of switching from prednisone to deflazacort aimed to improve benefit-risk. During 6-months average follow-up after switching in this uncontrolled study, physicians reported lower proportions of most adverse effects compared to the pre-switch period. Among the minority of charts with disease severity recorded, most showed stability of progressive disease.